Tagraxofusp
Not recommended HaematologyAuthority RequiredFirst-line line 💬 consumer voice
First-line treatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN) in patients who have not received prior systemic therapy.
1
Submissions
2023–23
On the record
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ICER range
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Cost basis
Decision on record
- Meeting Jul 2023 Not recommended Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)
From the public summary
PBAC outcome
The PBAC did not recommend tagraxofusp for the first line treatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN). The PBAC noted that BPDCN was a very rare disease with a poor prognosis, but considered that, based on the data presented, tagraxofusp did not demonstrate superiority over standard chemotherapy.PSD · Jul 2023
The primary reason for this outcome was due to the comparative clinical evidence presented.PSD · Jul 2023
Economic analysis
The submission presented a stepped economic evaluation of tagraxofusp compared to TPC in patients newly diagnosed with BPDCN based on data from treatment naïve patients receiving 12 mcg/kg tagraxofusp in Stages 1-3 of Study 0114 (henceforth referred to as Stages 1-3), and a matched indirect comparison with Laribi 2020.PSD · Jul 2023
The type of economic evaluation presented was a cost-utility analysis. Key components of the economic evaluation are presented in the table below. 31PSD · Jul 2023
Clinical claim
The submission described tagraxofusp as superior in terms of effectiveness, and superior in terms of safety compared to TPC. The ESC considered that these claims were poorly supported by the data presented in the submission.PSD · Jul 2023
The superior efficacy claim relied on an unanchored MAIC based on data from Study 0114 (a non-randomised study) for tagraxofusp and Laribi 2020 (a retrospective observational study) for TPC. There were no statistically significant differences for any of the outcomes compared in the MAIC.PSD · Jul 2023
Consumer comments
The PBAC noted the advice received via the Consumer Comments facility on the PBS website from Rare Cancers Australia supporting the tagraxofusp submission. The advice provided a patient perspective which described the benefits of tagraxofusp including the possibility of outpatient treatment, reduced side effects and the potential of bridging to HSCT. The PBAC noted that this advice was supportive of the evidence provided in the submission.PSD · Jul 2023
Financial management – risk sharing
No risk sharing arrangement was specified, but the sponsor indicated a willingness to negotiate one. For more detail on PBAC’s view, see section 7 PBAC outcome.PSD · Jul 2023
Cost-effectiveness
ICER values redacted; document text ends before economic analysis section is presented
Decision context
PopulationPatients with blastic plasmacytoid dendritic cell neoplasm (BPDCN) who have not received prior systemic therapy, with ECOG performance status of 2 or less.
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Jul 2023 | Not recommended | Treatment of Physician's Choice (TPC) consisting of AML-type, ALL-type, and lymphoma-type chemotherapy regimens | — | Single-arm · OS |
Consumer voice
Rare Cancers Australia provided consumer input supporting tagraxofusp, highlighting benefits including outpatient treatment, reduced side effects, and potential bridging to HSCT, which aligned with the clinical evidence presented.
The advice provided a patient perspective which described the benefits of tagraxofusp including the possibility of outpatient treatment, reduced side effects and the potential of bridging to HSCT. Consumer comments · PSD
treatment burdenquality of lifeaccess barriers